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UveitisPhase 2ACTHARNCT02931175

ACTH as A Re-emerging theRapy for Uveitis (The ACTHAR Study)

A Phase 2 study testing ACTH gel in people with Uveitis, run by Quan Dong Nguyen at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The study aims to evaluate the potential role of ACTH gel in the management of non-infectious uveitis. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Uveitis
What is being tested?
ACTH gel
Could I receive a placebo?
No, everyone receives an active treatment
How many people
28 participants
Where
7 sitesUnited States
Study dates
10 Nov 2017to 28 Oct 2021
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • In order to be eligible for the study, patients will be required to meet the criteria of 1 of the 3 following disease cohorts:
  • Active disease and are receiving no treatment. Active disease is defined as having at least 1+ Vitreous Haze using the Standardized Uveitis Nomenclature (SUN) Working Group scale and/or at least 1+ Vitreous Cell Count using Foster \& Vitale scale.
  • Active disease and are receiving prednisone ≥10 mg/day (or equivalent dose of another corticosteroid) or at least 1 other systemic immunosuppressant (all systemic immunosuppressants other than corticosteroids will be discontinued 30 days prior to the first administration of the study drug on Day 0). Patients receiving combination of prednisone ≥10 mg/day and at least one other systemic immunosuppressant are also eligible in this category.
  • Have inactive disease, defined as having <= 0.5+ Vitreous Haze OR <= 0.5+ Vitreous Cell Count (SUN scale), and are receiving prednisone ≥10 mg/day (or equivalent dose of another corticosteroid) or at least 1 other systemic immunosuppressant (all systemic immunosuppressants other than corticosteroids will be discontinued 30 days prior to the first administration of the study drug on Day 0). Patients receiving combination of prednisone ≥10 mg/day and at least one other systemic immunosuppressant are also eligible in this category.

Who cannot join

  • Subjects who have any of the following at the screening visit are not eligible for enrolment in this study:
  • Any significant ocular disease that could compromise vision in the study eye. These include, but are not limited to:
  • Diabetic retinopathy: proliferative diabetic retinopathy (PDR) or non-proliferative diabetic retinopathy (NPDR) that compromise the vision.
  • Age-related macular degeneration;
  • Myopic degeneration with active subfoveal choroidal neovascularization.
  • Advanced glaucoma status post trabeculectomy or tube/valve placement
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  • Any of the following treatments within 90 days prior to Day 0 or anticipated use of any of the following treatments to the study eye:
  • Intravitreal injections (including but not limited to steroids or anti-vascular endothelial growth factors);
  • Posterior subtenon's steroids.
  • Intraocular surgery within 90 days prior to Day 0 in the study eye;
  • Capsulotomy within 30 days prior to Day 0 in the study eye;
  • Any known ocular surgery (including cataract extraction or capsulotomy) of the study eye anticipated within the first 180 days following Day 0;
  • Intraocular pressure(IOP) ≥25 mmHg in the study eye (glaucoma patients maintained on no more than 2 topical medications with IOP <25 mmHg are allowed to participate);
  • Pupillary dilation inadequate for quality stereoscopic fundus photography in the study eye;
  • Media opacity that would limit clinical visualization;
  • Presence of any form of ocular malignancy in the study eye, including choroidal melanoma;
  • History of herpetic infection in the study eye or adnexa;
  • Presence of known active or inactive toxoplasmosis in either eye;
  • Presence of ocular or periocular infection in either eye;
  • Participation in other investigational drug or device clinical trials within 30 days prior to Day 0, or planning to participate in other investigational drug or device clinical trials within 180 days following Day 0. This includes both ocular and non-ocular clinical trials.
  • Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization.
  • Prior treatment with any cell-depleting therapies, including investigational agents or approved therapies, some examples are anti-cluster of differentiation (CD) 4, anti- cluster of differentiation (CD)5, anti-cluster of differentiation (CD) 3, anti-cluster of differentiation (CD)19 and anti-cluster of differentiation (CD)20.
  • Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of baseline.
  • Immunization with a live/attenuated vaccine within 4 weeks prior to baseline.
  • Previous treatment with ACTHAR within 3 months of day 0 of study visit.
  • Any previous treatment with alkylating agents such as chlorambucil, or with total lymphoid irradiation.
  • Exclusions for General Safety:
  • History of severe allergic or anaphylactic reactions to proteins of porcine origin.
  • Evidence of serious uncontrolled concomitant cardiovascular (including history of congestive heart failure, uncontrolled hypertension), nervous system (include myasthenia gravis), pulmonary (including obstructive pulmonary disease), renal, hepatic, endocrine (include uncontrolled diabetes mellitus, hypothyroidism), gastrointestinal disease (including history of or presence peptic ulcer disease, complicated diverticulitis, ulcerative colitis, or Crohn"s disease), Scleroderma or Osteoporosis.
  • Current liver disease as determined by principal investigator unless related to primary disease under investigation.
  • Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other infections (including but not limited to tuberculosis and atypical mycobacterial disease, Hepatitis B and C, and herpes zoster, but excluding fungal infections of nail beds).
  • Any major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening.
  • Active Tuberculosis (TB) requiring treatment within the previous 3 years. Patients should be evaluated for latent and/or active TB within one month of the screening as part of the evaluation by the investigator to rule out infectious uveitis before referring the patient to the study. If positive, patients should be managed following local practice guidelines prior to initiating H.P. ACTHAR gel. Patients treated for TB with no recurrence in 3 years are permitted.
  • Primary or secondary immunodeficiency (history of or currently active)
  • Evidence of active malignant disease, malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors, except basal and squamous cell carcinoma of the skin or carcinoma in-situ of the cervix uteri that has been excised and cured)
  • Pregnant women or nursing (breast feeding) mothers.
  • Patients with reproductive potential not willing to use an effective method of contraception.
  • History of alcohol, drug or chemical abuse within 1 year prior to screening.
  • Neuropathies or other conditions that might interfere with pain evaluation unless related to primary disease under investigation.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Retina Consultants of Houston

    Bellaire, Texas, 77401

  • Retina Centers Professional Corporation - RCPC

    Cleveland, Ohio, 44139

  • Texas Retina Assiciates

    Dallas, Texas, 75231

  • TExas Retina Associates

    Dallas, Texas, 75231

  • Retina Vitreous Associates, Medical Group

    Los Angeles, California, 90017

  • Stanford University

    Palo Alto, California, 94303

  • Metropolitan Eye Research and surgery Institute

    Waltham, Massachusetts, 02451

Other trials in United States

About this study

Official title: An Open-label, Multi-center, Randomized, Phase II Study of the Safety,Efficacy and Bioactivity of Two Dose Regimens of Subcutaneous Injections of ACTH Gel in Patients With Non-infectious Uveitis

The study aims to evaluate the potential role of ACTH gel in the management of non-infectious uveitis.

Read the detailed description

Uveitis is a term used to describe a group of inflammatory disorders involving the uveal tract (iris, ciliary body and choroid). It is amongst the most common causes in the list of preventable cause of blindness in the developed world.

H.P. Acthar Gel is a highly purified sterile preparation of the adrenocorticotropic hormone (ACTH) gelatin to provide a prolonged release after intramuscular or subcutaneous (SC) injection.

ACTH is part of a group of molecules called melanocortins (MC) (ACTH, α, β, γ-MSH) that are produced, in human bodies, by breakdown of a common larger precursor called proopiomelanocortin (POMC). A very well-known anti-inflammatory mechanism of action of ACTH is the production glucocorticoids by stimulating the adrenal glands. ACTH has also been shown to bind with all five melanocortin receptors (MCRs). MCRs have a variety of roles including cortisol production and regulation of immune modulation by ACTH.

ACTHAR is an open-label, multi-center, randomized, phase II study to evaluate the effect of two dose regimens of repeated SC injections of ACTH gel in patients with active non-infectious intermediate, posterior, or pan-uveitis followed over a period of 12 months.

ACTHAR study will be conducted at up to 7 clinical sites in USA. The study will be coordinated by the Ocular Imaging Research and Reading Center (OIRRC), which will serve as the coordinating and reading center for the ACTHAR Study.

The primary endpoint of the study will be at month 6, with an active, as-needed treatment extension phase from month 6 to month 12.

Thirty-six (36) patients with non-infectious intermediate, posterior, or pan-uveitis will be enrolled and randomized (1:1) to one of the two treatment arms:

  1. Mandatory twice a week (Mondays and Thursdays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.
  2. Mandatory thrice a week (Mondays, Wednesdays, and Fridays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.

Starting at month 6, retreatment will be offered to study subjects who have demonstrated any level of response during the first 6 months and who meet any of these Retreatment Criteria listed below. Patients receiving retreatment will receive the dose that was assigned to them at randomization.

Conditions
Uveitis
Being tested
ACTH gel
Keywords
ACTH gel, Non-Infectious Uveitis, ACTHAR, Posterior Uveitis, Intermediate Uveitis, Pan Uveitis, Uveitis
Medical subject headings
Uveitis, Uveitis, Posterior, Uveitis, Intermediate

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ACTH gelDrug

    Also called Corticotropin

    Starting at month 6, retreatment will be offered to study subjects who have demonstrated any level of response during the first 6 months and who meet any of these Retreatment Criteria listed below. Patients receiving retreatment will receive the dose that was assigned to them at randomization.

Study groups in NCT02931175
Study groupRoleWhat they receive
Group 1ExperimentalMandatory twice a week (Mondays and Thursdays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.
Receives: Drug: ACTH gel
Group 2ExperimentalMandatory thrice a week (Mondays, Wednesdays, and Fridays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.
Receives: Drug: ACTH gel

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of ocular adverse events (AEs)

    The incidence and severity of ocular adverse events, as identified by eye examination.

    Measured: Month 6

  2. Incidence of systemic adverse events (AEs)

    The incidence and severity of systemic adverse events.

    Measured: Month 6

Secondary outcomes (16)

  1. Mean change from Baseline (BL) in vitreous haze (VH)

    Mean change from BL in vitreous haze using the Standardization of Uveitis Nomenclature (SUN) scale.

    Measured: Month 6

  2. Proportion of patients achieving a ≥ 1 and ≥ 2 step improvement or resolution of VH

    The proportion of patients achieving a ≥ 1 and ≥ 2 step improvement or resolution of VH on SUN scale after therapy or at time of rescue.

    Measured: Month 3, 6, 12

  3. Mean change from BL in graded anterior chamber cells

    Mean change from BL in graded anterior chamber cells after therapy or at time of rescue.

    Measured: Month 3, 6, 12

  4. Mean change from BL in Best Corrected Visual Acuity (BCVA)

    Mean change from BL in BCVA using the ETDRS

    Measured: Month 3, 6, 12

  5. Mean change in degree of inflammation

    Mean change in degree of intermediate uveitis, retinal vasculitis, chorioretinitis, or other types of inflammation as documented at baseline compared to evaluation at month 6 and 12 after therapy or at time of rescue.

    Measured: Month 3, 6, 12

  6. Mean change in leakage of disc.

    Mean change in leakage of disc

    Measured: Month 3,6, 12

  7. Change from Baseline in macular thickness

    Change from Baseline in macular thickness as measured by Spectral Domain Optical Coherence Tomography (SD-OCT).

    Measured: Month 3, 6, 12

  8. The proportion of patients who achieve a complete response in the study eye and discontinue prednisone.

    The proportion of patients who achieve a complete response in the study eye and discontinue prednisone.

    Measured: Month 3, 6, 12

  9. The proportion of patients who achieve a partial response in the study eye and discontinue prednisone.

    The proportion of patients who achieve a partial response in the study eye and discontinue prednisone.

    Measured: Month 3, 6, 12

  10. Proportion of patients achieving a ≥ 1 and ≥ 2 step improvement or resolution of VH

    The proportion of patients achieving a ≥ 1 and ≥ 2 step improvement or resolution of VH on SUN scale after therapy or at time of rescue.

    Measured: Month 12

  11. Mean change from BL in graded vitreous cells

    Mean change from BL in graded vitreous cells after therapy or at time of rescue.

    Measured: Month 3, 6, 12

  12. Mean change from BL in aqueous flare

    Mean change from BL in aqueous flare as measured by aqueous flare meter.

    Measured: Month 3, 6 12

  13. Mean change in leakage of vasculature

    Mean change in leakage of vasculature

    Measured: Month 3,6, 12

  14. Mean change in leakage of chorioretinal lesions

    Mean change in leakage of chorioretinal lesions

    Measured: Month 3, 6, 12

  15. Mean change in Leakage in Macula

    Mean change in Leakage in Macula

    Measured: Month 3, 6, 12

  16. Changes in OCTA

    Changes in OCTA at various time points

    Measured: Months 3, 6, 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Oct 2016 (estimated)
  2. Study start10 Nov 2017
  3. Primary completion3 Apr 2020
  4. Study completion28 Oct 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
28 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02931175
Sponsor's protocol ID
ACTHAR-01
Acronym
ACTHAR
Status last verified
Sep 2018
First submitted
6 Oct 2016
Last updated
17 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Sadiq MA, Agarwal A, Hassan M, Afridi R, Sarwar S, Soliman MK, Do DV, Nguyen QD. Therapies in Development for Non-Infectious Uveitis. Curr Mol Med. 2015;15(6):565-77. doi: 10.2174/1566524015666150731103847.

    PubMed 26238367

  • Montero-Melendez T. ACTH: The forgotten therapy. Semin Immunol. 2015 May;27(3):216-26. doi: 10.1016/j.smim.2015.02.003. Epub 2015 Feb 26.

    PubMed 25726511

  • Durrani OM, Meads CA, Murray PI. Uveitis: a potentially blinding disease. Ophthalmologica. 2004 Jul-Aug;218(4):223-36. doi: 10.1159/000078612.

    PubMed 15258410

  • Lee K, Bajwa A, Freitas-Neto CA, Metzinger JL, Wentworth BA, Foster CS. A comprehensive review and update on the non-biologic treatment of adult noninfectious uveitis: part I. Expert Opin Pharmacother. 2014 Oct;15(15):2141-54. doi: 10.1517/14656566.2014.948417.

    PubMed 25226529

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ACTH gel in people with Uveitis, run by Quan Dong Nguyen at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of ocular adverse events (AEs) (time frame: Month 6). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02931175, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025.

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